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Gene therapy decreases seizures in a model of Incontinentia pigmenti

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Incontinentia pigmenti (IP) is a genetic disease leading to severe neurological symptoms, such as epileptic seizures, but no specific treatment is available. IP is caused by pathogenic variants that inactivate… Click to show full abstract

Incontinentia pigmenti (IP) is a genetic disease leading to severe neurological symptoms, such as epileptic seizures, but no specific treatment is available. IP is caused by pathogenic variants that inactivate the Nemo gene. Replacing Nemo through gene therapy might provide therapeutic benefits.

Keywords: therapy decreases; gene therapy; incontinentia pigmenti; gene; decreases seizures

Journal Title: Annals of Neurology
Year Published: 2017

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